Melanoma/Skin Cancer Health Center

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New Drug May Treat Advanced Melanoma

No Cure, but Study Shows New Melanoma Drug Far Better Than Standard Treatment

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What Should Today's Melanoma Patients Do?

Despite all this excitement, the reality is that PLX4032 is not yet ready for prime time. A phase III clinical trial is now enrolling patients with BRAF-positive tumors. In that trial, some patients will get the drug (in a version dubbed RO5185426) and others will get standard treatment.

Only after this trial is completed and analyzed will it be known whether the drug truly works -- and only then will it be available to patients.

There's a bit more good news for the future. A similar drug from GlaxoSmithKline, called GSK2118436, is enrolling patients in a phase II study.

"The bottom line is that if I were a patient, I would be looking for either one in a clinical trial," Flaherty says.

Another big question is what the new drug might mean for patients with early-stage melanoma. Stage I tumors can be cured with surgery. But melanoma is infamous for how quickly it can spread through the body from a small skin tumor. About half of patients with metastatic melanoma had previously had apparently curative melanoma surgery.

It's not at all clear whether giving one of the new BRAF inhibitors to such patients would keep their cancers from coming back.

"Getting to the clinical trial evaluation of this is urgent and we are getting that under way," Flaherty says. "We don't know if eradicating microscopic cancer cells is the same trick as beating down large tumors. But with two other targeted therapies, Gleevec and Herceptin, it does appear that they truly can prevent recurrences. We are just as hopeful for this drug."